| dc.description.abstract | Assessing the cost effectiveness for drugs targeting ultra-rare diseases is complex
given the limited clinical evidence for many drugs, yet is growing in importance as the
number of these drugs being commercialized is increasing rapidly. Selumetinib is a
recently FDA-approved MEK inhibitor for patients aged 3 to 18, with Neurofibromatosis
Type I (NF1) Inoperable Plexiform Neurofibromas (PN), a rare genetic disorder, and has
been shown in clinical trials to stabilize and contract tumors by up to 20%. As this is an
expensive drug with costs ranging from $76,672.03 to $357,802.84CAD (2022) per year
of treatment depending on one’s body-surface-area, examining its cost-effectiveness to
the health system is important; yet made difficult due to the behaviour of the disease
largely dependent on one’s age. Accordingly, this paper uses the best available clinical
evidence, including that of the recent SPRINT clinical trial, to produce a time-dependent
Markov model, where costs, utilities and probabilities are based on either the Markov
stage, patient age, or both; to evaluate the cost-effectiveness of potential dosing
strategies of Selumetinib for NF1 Inoperable PN patients aged 3 to 18. Potential treatment
strategies were developed, differing on the number of years a patient would take
Selumetinib following clinical response. Incremental cost effectiveness ratios (ICER),
representing cost per quality adjusted life year (QALY), were calculated for the strategies,
which ranged from $74,671.90 CAD to $255,674.71 CAD. A Monte-Carlo simulation was
run in each scenario, assuming a maximum willingness-to-pay threshold of $150,000,
which revealed Selumetinib being cost-effective in 70.94% of iterations in the lowest
intensity strategy, and in only 6.717% of iterations of the highest intensity. Recognizing
this study is limited to understanding the cost-effectiveness of potential treatment strategies of this drug for this population, this paper hopes to support development of
such strategies, along with clinical and epidemiological evidence, in providing an
economic perspective. Beyond this application, this paper sets the stage not only for
further cost-effectiveness of Selumetinib for other populations, but also of other novel
drugs with costs, utilities, and probabilities, in part, based on patient age, through use of
the time-dependent Markov model. Future research directions are discussed, based on
the limitations of the current study. | en_US |
